Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs.
Saved in:
| Title: | Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs. |
|---|---|
| Authors: | Whittaker MN; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA, USA., Testa LC; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA., Quigley A; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA., Brooks DL; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA., Grandinette SA; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA., Said H; Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA, USA.; Department of Bioengineering, George Mason University, Fairfax, VA, USA., Dwivedi G; Division of Infectious Diseases, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.; Penn Institute for RNA Innovation, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Jindal I; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA., Volpp D; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA., Hacker JL; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA., Qu P; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA., Wang JZ; Agilent Technologies Inc., Santa Clara, CA, USA., Levine MA; Division of Endocrinology and Diabetes, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Ahrens-Nicklas RC; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Division of Human Genetics and Metabolism, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Li Q; Department of Biochemistry and Molecular Biology, Sidney Kimmel Medical College, Jefferson Institute of Molecular Medicine, Thomas Jefferson University, Philadelphia, PA, USA.; PXE International Center of Excellence in Research and Clinical Care, Thomas Jefferson University, Philadelphia, PA, USA., Musunuru K; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA. kiranmusunuru@gmail.com.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA. kiranmusunuru@gmail.com.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA. kiranmusunuru@gmail.com.; Division of Human Genetics and Metabolism, Children's Hospital of Philadelphia, Philadelphia, PA, USA. kiranmusunuru@gmail.com., Alameh MG; Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA, USA. mg.alameh@pennmedicine.upenn.edu.; Department of Bioengineering, George Mason University, Fairfax, VA, USA. mg.alameh@pennmedicine.upenn.edu.; Penn Institute for RNA Innovation, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. mg.alameh@pennmedicine.upenn.edu.; Department of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA. mg.alameh@pennmedicine.upenn.edu., Peranteau WH; The Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA. peranteauw@chop.edu.; Division of Pediatric General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, USA. peranteauw@chop.edu., Wang X; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA. xiao8@pennmedicine.upenn.edu.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA. xiao8@pennmedicine.upenn.edu.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA. xiao8@pennmedicine.upenn.edu. |
| Source: | Nature biomedical engineering [Nat Biomed Eng] 2025 Oct 28. Date of Electronic Publication: 2025 Oct 28. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Springer Nature Country of Publication: England NLM ID: 101696896 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2157-846X (Electronic) Linking ISSN: 2157846X NLM ISO Abbreviation: Nat Biomed Eng Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
Be the first to leave a comment!
